Allogeneic hematopoietic stem cell transplantation for patients with hemophagocytic syndrome (HPS) in Japan

Seventeen cases (age at onset, 1 month to 18 years; M/F, 9/8) of hemophagoc ytic syndrome which received allogeneic hematopoietic stem cell transplanta tion (SCT) in Japan during the period 1988-1998 are reported. The patients consisted of six familial inheritance-proven erythrophagocytic lymphohistio cytosis (FEL), five familial inheritance-unknown and infective agents-unkno wn HLH (of which two were highly likely to have been FEL with characteristi c CNS signs), and six aggressive Epstein-Barr virus (EBV)-related HLH (of w hich two were natural killer cell-type large granular leukemia/lymphoma-ass ociated hemophagocytic syndrome, EBV-NK-LGLL-HPS). All cases were treated i ntensively with immuno-chemotherapy, or with chemotherapy before SCT, As so urces of SCT, 12 cases received bone marrow cells (sibling six, father one, URD five), two cord blood, two purified CD34-positive cells, and one PBSC, SCTs were successful in all 17 cases, apart from one receiving CD34-positi ve SCT. Following SCT, four patients relapsed and five died with a median f ollow-up of 23 months. Among the relapsed cases, the two EBV-NK-LGLL-HPS pr eviously published as successfully transplanted were included. Among the fa tal cases, three patients died from relapsed active disease and the remaini ng two from fatal post-SCT EBV-positive T cell lymphoma and extensive chron ic GVHD, respectively. As of the end of September 1998, 10 patients are ali ve without disease for 3.5 months to 147 months, while two post-SCT patient s are still having therapy for residual/recurrent disease. The Kaplan-Meier analysis showed a 2-year event-free survival after SCT as 54.0 +/- 13.0%.